Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study
酶替代疗法
医学
内科学
免疫原性
糖原贮积病Ⅱ型
疾病
临床终点
临床试验
免疫学
免疫系统
作者
Si Houn Hahn,David Kronn,Nancy D. Leslie,Loren Peña,Pranoot Tanpaiboon,Michael J. Gambello,James B. Gibson,Richard E. Hillman,David W. Stockton,John Day,Raymond Wang,Kristina An Haack,Raheel Shafi,Susan Sparks,Yang Zhao,Catherine Wilson,Priya S. Kishnani
出处
期刊：Genetics in Medicine [Springer Nature] 日期：2018-03-22卷期号：20 (10): 1284-1294被引量：29
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(2025-6-4)
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